Pediatric Blood & Cancer最新文献

Background

Delays in diagnosing pediatric brain tumors are often associated with limited awareness of the signs and symptoms by parents and healthcare professionals, as well as the absence of routine childcare follow-ups and delays in healthcare system referrals.

Aims

To explore opportunities for reducing diagnostic delays, the study assessed the knowledge of pediatric brain tumor signs and symptoms, routine follow-up care for children, use of the Child Health Book (CHB), and referral intervals of a suspected case to a specialized center.

Procedure

This cross-sectional study collected data through interviews and virtual questionnaires.

Results

Between August and November 2023, 200 parents (pediatric and oncology departments) and 147 healthcare professionals (primary and tertiary care) participated. Except for headaches and seizures, the rates of parental recognition of warning signs were below 70%. Physicians in tertiary care demonstrated greater recognition of these warning signs than those in primary care (p = 0.011). Recognition rates among nurses were below 75%. Primary and tertiary care professionals reported referral intervals >1 month in 10%–15% cases. Children routine follow-up care was reported in both levels. Over 75% of all participants reported that the CHB could be a useful tool for educating about childhood cancer.

Conclusions

Our study provides essential insights to improve the early diagnosis of pediatric brain tumors. The findings emphasize the need to strengthen pediatric care follow-ups and use of CHB by parents and healthcare professionals to raise awareness of warning signs and symptoms, along with a flowchart for timely and accurate referrals to specialized centers.

Background

Recent research elucidated the prognostic significance of molecular biology in Wilms tumor (WT) by linking somatic genomic variants (such as gain of chromosome 1q) to unfavorable patient outcomes. This analysis describes the clinical impact of copy number variations (CNV) in tumor samples of WT patients with stage IV disease.

Methods

Tumor samples of 55 WT patients with stage IV disease from the United Kingdom, France, and Germany enrolled in the SIOP 2001 study and treated with preoperative chemotherapy (pCHT) were examined for their CNVs of chromosome 1q and other regions of interest using multiplex ligation-dependent probe amplification (MLPA). The identified CNV were analyzed regarding their prognostic impact.

Results

Chromosome 1q gain (1q+) and TP53 loss occurred in 38.2% and 16.4% of tumors and were associated with older patient age at diagnosis (median [months]: 65 and 64 vs. 49 each, p = 0.03 and 0.02, respectively) and poorer 5-year event-free survival (40.0% and 11.1% vs. 67.7% and 82.6%, p = 0.04 and <0.01, respectively) compared to their specific control group of tumors without the respective CNV. In patients with pulmonary-only metastasis, 1q+ was an adverse prognostic marker irrespective of remission status after pCHT with or without metastasectomy. A simultaneous MYCN gain occurred more frequently in tumors with 1q+ than in tumors without 1q+ (p = 0.03). TP53 loss was linked to high-risk histology and inferior 5-year overall survival (p < 0.001).

Conclusions

We confirm the prognostic relevance of 1q+ and TP53 loss in stage IV WTs and emphasize their potential utility for future treatment stratification.

Background and Aims

Pediatric salivary gland malignancies (SGM) present challenges in managing cervical nodes. We aimed to characterize lymph node invasion to inform decisions regarding the need of systematic wide lymph node dissection (WLND).

Methods

International retrospective study, conducted across seven large French and American pediatric centers, including pediatric patients (0–18 years) diagnosed with SGM from 2000 to 2020.

Results

Among the 82 patients (median age 13 years), the parotid gland was frequently affected (60 cases). Histotypes comprised mucoepidermoid (mucoepidermoid carcinoma [MEC], 43 cases), acinic cells (acinic cells carcinoma [AcCC], 22 cases), adenoid cystic (adenoid cystic carcinoma [AdCC], 8 cases), (MASC, 6 cases), and adenocarcinoma (3 cases). Primary treatments were surgery (82 cases) and radiotherapy (20 cases; median dosage 64 gray). Cervical nodes therapy included WLND (≥2 levels, 29 cases), limited nodes resection (LNR; one level, 13 cases), and/or irradiation (4 cases; median 54 gray; range 52.0–63.0). At diagnosis, six patients had cervical node invasion (CNI) managed with LNR (four cases), WLND (two cases), and radiotherapy (three cases). After a median follow-up of 6 years (range 1–22), nine patients had tumor event: local (four cases), cervical relapse/progression (three cases) or combined (two cases). Of the nine with CNI, at diagnosis or relapse, four had MASC. Five-year event-free and overall survival (OS) rates were, respectively, 90.1% and 98.8%.

Conclusions

CNI is rare in pediatric SGM but noted in 11% of cases, with higher incidence in MASC. Overall, outcome in SGM is good with a tailored locoregional multidisciplinary approach. Systematic lymph node dissection should be reconsidered.

SUMMARY

This international multi-institutional study analyzed the clinical presentation and the cervical pattern of relapse of 82 pediatric patients with newly diagnosed salivary gland malignancies. Overall, nodal invasion was rare at diagnosis and only noted in 7%. In addition, 6% developed nodal relapse during follow-up. Incidence of nodal spread was frequent in mammary analogue secretory carcinoma (MASC). The overall outcome was promising with a tailored locoregional multidisciplinary approach. Systematic lymph node dissection should be reconsidered in pediatric salivary gland tumors.

Background

A pediatric cancer diagnosis is overwhelming and stressful for the whole family. Patient-centered communication during the diagnostic conversation can support medical and psychosocial adaptation to the disease. Treatment of pediatric leukemia has become increasingly complex and requires a specific skillset from clinicians in effectively conveying information to families. The objective of the current study was to gain insight in the experiences and perspectives of pediatric oncologists when communicating leukemia diagnoses to families.

Procedure

In this exploratory qualitative study, oncologists were eligible to participate for each diagnostic conversation between May 2022 and February 2023 of families participating in a larger study. Twenty-six semi-structed interviews with 16 oncologists were thematically analyzed.

Results

Two interrelated conversational goals were identified: (i) informing the family about the illness, prognosis, and treatment; and (ii) creating trust and comfort for the family implying they are in the right place for maximal chance of survival. Oncologists experienced a challenge in balancing a high amount of information provision in a short timespan with simultaneously monitoring the (emotional) capacity and needs of the family to process information. Remarkably, oncologists commonly seem to rely on intuition to guide the family through the diagnostic conversation. They mentioned to sometimes postpone answering to family-specific informational needs and prioritized information they assume to be more helpful for the family at that time.

Conclusions

During diagnostic conversations, oncologists aim to convey information they assume supports the needs of the family. Future research should investigate how these communication strategies are perceived by families.

Background

Surgery remains the cornerstone of treatment for rhabdomyosarcoma (RMS) in children. However, there is considerable variation in surgical management practices worldwide, highlighting the need for standardized Clinical Practice Guidelines (CPG).

Methods

The CPG development involved assembling a multidisciplinary group, prioritizing 10 key topic areas, conducting evidence searches, and synthesizing findings. Recommendations were voted on using the GRADE (Grading of Recommendations, Assessment, Development, and Evaluations) methodology.

Recommendations

The panel recommended regional lymph node evaluation for patients with paratesticular RMS who are more than 10 years old and extremity RMS. Other suggestions included pretreatment re-excision for incompletely resected RMS, preoperative radiation therapy for unresectable tumors, maintaining a 0.5 cm resection margin, and tumor bed marking with surgical clips. The panel also suggests resection of residual metastatic disease following chemotherapy, resection of relapsed disease, and the least invasive approach for managing patients presenting with obstruction.

Conclusion

This CPG provides evidence-based surgical management recommendations for RMS that can be adapted to diverse resource settings.

Background

Hydration and urine alkalinization are the mainstays for the prevention of methotrexate-induced nephrotoxicity. Current oncology protocols recommend pediatric patients who are administered high-dose methotrexate (HDMTX) to be aggressively hydrated with an alkaline solution, which may lead to overhydration. This pilot study sought to determine whether reduced posthydration results in a shorter time to methotrexate elimination without increasing adverse effects.

Methods

A prospective randomized controlled crossover study design of pediatric patients with acute lymphoblastic leukemia was performed. Patients were randomized to begin with standard or reduced volume intravenous fluids. Over the course of four cycles of HDMTX, patients alternated between the standard rate of 125 mL/m²/h and a reduced volume rate of 62.5 mL/m²/h. The primary endpoint was the time from the start of HDMTX to a serum methotrexate concentration less than 0.1 µmol/L.

Results

Data from 37 HDMTX courses were analyzed in 10 patients aged 1–17 years. The median time to methotrexate elimination was similar between the standard and reduced hydration regimens at 71.7 h (60.8–115.6 h) versus 72.9 h (59.9–132 h, p value = 0.6539). There was no difference in the change from baseline to maximum creatinine (10% vs. 18.9%, p value = 0.6566), maximum weight gain (0.7 kg vs. 0.4 kg, p value = 0.0967), or rates of severe mucositis between hydration regimens.

Conclusion

Reduced posthydration appeared to be safe and provided similar time to HDMTX elimination. A multicenter study is indicated to confirm the use of reduced hydration to optimize supportive care in pediatric patients administered HDMTX.

Trial Registration: NCT03964259.

Background

Systemic inflammatory diseases (SIDs) have been reported in patients with sickle cell disease (SCD), but clinical data in children are scarce.

Objectives

To identify clinical and laboratory features at diagnosis of SID in children with SCD and to describe their evolution.

Methods

Data from children with SCD and SIDs were retrospectively collected in a French multicenter study from 1991 to 2018. Information included clinical characteristics, inflammatory markers, autoantibodies patterns, treatments, and complications. Inflammatory marker levels were compared at SID diagnosis and at the last follow-up. Statistical analyses were performed using Cran R software.

Results

Among a cohort of 3800 children with SCD, 43 SIDs were identified in 35 study participants: autoimmune liver disease (AILD, n = 13), inflammatory bowel disease (IBD, n = 7), juvenile idiopathic arthritis (JIA, n = 6), systemic lupus erythematosus (n = 4), autoimmune hemolytic anemia (n = 3), Sjögren syndrome (n = 1), histiocytic necrotizing lymphadenitis (n = 2), vasculitis (n = 2), myasthenia gravis (n = 1), sarcoidosis (n = 1), idiopathic inflammatory granulomatous uveitis (n = 1), mixed connective tissue disease (n = 2). Prevalence of SID was 0.9% in our cohort of children with SCD. The median time between initial symptoms and SID diagnosis was 10 (3–20) months, notably longer in children with JIA, IBD, and Sjögren syndrome. Sixteen patients (46%) exhibited hypergammaglobulinemia (>20 g/L) at diagnosis. No significant differences were observed for other inflammatory parameters. Twenty-one children (60%) received systemic steroids and 13 (37%) biological therapies. Three patients (9%) underwent hematopoietic stem cell transplantation. Nine patients (26%) had severe infections; one died.

Conclusion

Delayed diagnosis was frequent due to overlapping clinical presentations between SCD and SID. Clinicians must be aware of warning signs associated with elevated inflammatory markers, hypergammaglobulinemia, or specific antibodies. Therapeutic strategies remain challenging.